Looming FDA Decisions Could Swell Number of Gene Therapies on Market

by Leslie Small

 

In its latest drug pipeline report, UnitedHealth Group-owned PBM Optum Rx takes a look at two high-priced gene therapies that are slated for FDA approval soon. Both drugs highlighted in the report, Zynteglo and Skysona, are manufactured by bluebird bio, Inc., a Massachusetts-based biotech company. An FDA advisory panel in June recommended that they be approved.    Landmark therapies extract cells from patients  The FDA is expected to make its final decision by Aug. 19 on Zynteglo, which treats transfusion-dependent beta-thalassemia — a disorder caused by genetic mutations in cells that carry oxygen throughout the body.   Of the roughly 3,000 beta-thalassemia patients in the U.S., about half are dependent on regular red blood cell transfusions. The only current cure for the condition is stem cell transplantation from a matched donor, but fewer than 25% of patients have access to a suitable donor match.  Zynteglo, however, could cure beta-thalassemia through a one-time intravenous infusion of a patient’s own modified stem cells following chemotherapy conditioning.  Skysona, meanwhile, treats a condition in young boys known as cerebral adrenoleukodystrophy (CALD), which can cause disability and early death in patients who lack a protein called ALDP needed to break down fatty acids. There are about 40 CALD patients in the U.S. per year, and most will die within a decade of diagnosis if they don’t receive a bone marrow stem cell transplant, per the Optum report.    Like Zynteglo, Skysona works by extracting cells from a patient — in this case, bone marrow cells — modifying them with a virus, and reinfusing them back into the patient.   Therapy prices may top $2M  To cope with the high cost of gene therapies, some payers are experimenting with strategies such as outcomes-based contracts and reinsurance.  Both Zynteglo and Skysona could cost around $2.1 million for a one-time dose, the Optum report noted.   Zynteglo and Skysona aren’t the only gene therapies that bluebird has in the works. The firm’s sickle cell disease treatment, lovotibeglogene autotemcel — as well as Vertex/CRISPR Therapeutics’s exagamglogene autotemcel — could both be filed with the FDA by end of 2022 or the first quarter of 2023, according to Arash Sadeghi, clinical pharmacist, Pipeline & Drug Surveillance, Optum Rx.  Sickle cell disease is a key area of interest for gene-therapy-focused firms “because of the large target population relative to other orphan conditions,” he tells AIS Health, a division of MMIT.

 

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